Two approvals, an Orphan Drug designation in the week ending April 12

Regulatory Updates

The FDA approved Amgen/UCB’s romosozumab (Evenity) on 4/9/2019 for the treatment of osteoporosis in postmenopausal women at high risk for fracture. The drug has a Boxed Warning regarding the increased risk of myocardial infarction, stroke and cardiovascular death. Amgen set WAC for romosozumab at $1,825 a month for an annual cost of $21,900. Romosozumab will likely join abaloparatide and teriparatide as a specialty drug for the treatment of osteoporosis. WAC for abaloparatide is $22,000, while the Federal Supply Schedule price is $11,800. For teriparatide, WAC is $36,000, and the Federal Supply Schedule price is $26,000. ICER estimated the cost per quality adjusted life year gained over the bisphosphonate zoledronic acid as $334,000 for abaloparatide and $942,000 for teriparatide. Due to the delay in FDA review, ICER did not include rombosozumab in its review.

The FDA approved Janssen’s erdafitinib on 4/12/2019 for the treatment locally advanced or metastatic bladder cancer with fibroblast growth factor receptor (FGFR)3 or FGFR2 mutations who have progressed on platinum-containing chemotherapy. A companion lab test was also approved to identify patients with these mutations. WAC for erdafitinib will be $10,080 to $22,680 for a 28-day supply, depending on dose for an annual cost of $131,040 to $294,840.

Celgene and Acceleron submitted a BLA for luspatercept in April 2019.

The FDA designated FibroGen’s pamrevlumab an Orphan Drug for the treatment of Duchenne muscular dystrophy (DMD) in April 2019.

Ghana has accepted 400,000 doses of GSK’s Mosquirix to pilot the malaria vaccine.

Remimazolam is an investigational benzodiazepine whose effects can be reversed with flumazenil. Cosmo Pharmaceuticals licensed US marketing rights for remimazolam from Paion. Cosmo submitted an NDA for remimazolam in April 2019.

New Trial Results Announced

Poxel announced that in a 24-week, 213 patient, Phase III, TIMES 1 trial, imeglimin 1,000 mg lowered HbA1c 0.87% more than placebo. 

New Published Trial Results

The Phase III APECS trial was terminated after an interim analysis showed no improvement with Merck’s verubecestat in the Clinical Dementia Rating Scale-Sum of Boxes compared to placebo in patients with prodromal Alzheimer's Disease. There were 1,454 patients enrolled in the trial at termination and 704 completed the 104-week study period.

In a 12-week, 160 patient, Phase II trial, treatment with Merck’s ruzasvir and uprifosbuvir (no grazoprevir) resulted in SVR in 52/54 genotype 1a patients, 15/15 genotype 1b patients, 28/29 with genotype 2, 30/39 with genotype 3, 18/20 with genotype 4 and 2/3 with genotype 6 in patients chronically infected with HCV genotypes 1-6.